Johannes Oldenburg, Martin Olivieri, Songkai Yan, Ying Yang, Radovan Tomic, Xiang Zhang, Douglass Drelich, Natalie Jakobs, Mariasanta Napolitano
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These outcomes were also explored in PwHB with pre- and post-rIX-FP switch data.</p><h3>Results</h3><p>Of 194 PwHB, 107 and 87 received rIX-FP and rFIXFc prophylaxis, respectively. The mean FIX consumption of rIX-FP was significantly lower compared to rFIXFc (42.4 vs. 65.2 IU/kg/week, <i>p</i> = 0.0001), with mean dosing intervals of 9.5 days (rIX-FP) and 7.9 days (rFIXFc). The mean bleeding rates for rIX-FP versus rFIXFc, respectively, were: ABR 0.7 versus 1.1 (<i>p</i> = 0.6704), AsBR 0.1 versus 0.3 (<i>p</i> = 0.3427), and AjBR 0.3 versus 0.4 (<i>p</i> = 0.5296). Subgroup analyses for PwHB with severe and moderate hemophilia B separately showed similar numerical patterns when comparing these outcomes. In the 18 patients with switch data, a significant reduction in FIX consumption was observed (median 51.7 to 33.3 IU/kg/week, <i>p</i> = 0.0069), and the mean dosing interval was extended (7.2–9.5 days). The ABR (median 1.6–0.0, <i>p</i> = 0.0172; <i>n</i> = 18) and AjBR (median 0.6–0.0, <i>p</i> = 0.0200; <i>n</i> = 14) decreased significantly, while the AsBR decreased but not significantly (median 0.2–0.0, <i>p</i> = 0.1460; <i>n</i> = 14).</p><h3>Conclusion</h3><p>rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 9","pages":"4583 - 4596"},"PeriodicalIF":4.0000,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03303-7.pdf","citationCount":"0","resultStr":"{\"title\":\"Real-World Prophylaxis Outcomes with rIX-FP and rFIXFc for Males with Hemophilia B: Pooled Analysis of Medical Chart Data from Germany and Italy\",\"authors\":\"Johannes Oldenburg, Martin Olivieri, Songkai Yan, Ying Yang, Radovan Tomic, Xiang Zhang, Douglass Drelich, Natalie Jakobs, Mariasanta Napolitano\",\"doi\":\"10.1007/s12325-025-03303-7\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Introduction</h3><p>The current standard of care for people with severe hemophilia B is prophylaxis with factor IX (FIX) products. 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The mean bleeding rates for rIX-FP versus rFIXFc, respectively, were: ABR 0.7 versus 1.1 (<i>p</i> = 0.6704), AsBR 0.1 versus 0.3 (<i>p</i> = 0.3427), and AjBR 0.3 versus 0.4 (<i>p</i> = 0.5296). Subgroup analyses for PwHB with severe and moderate hemophilia B separately showed similar numerical patterns when comparing these outcomes. In the 18 patients with switch data, a significant reduction in FIX consumption was observed (median 51.7 to 33.3 IU/kg/week, <i>p</i> = 0.0069), and the mean dosing interval was extended (7.2–9.5 days). The ABR (median 1.6–0.0, <i>p</i> = 0.0172; <i>n</i> = 18) and AjBR (median 0.6–0.0, <i>p</i> = 0.0200; <i>n</i> = 14) decreased significantly, while the AsBR decreased but not significantly (median 0.2–0.0, <i>p</i> = 0.1460; <i>n</i> = 14).</p><h3>Conclusion</h3><p>rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. 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引用次数: 0
摘要
目前严重B型血友病患者的护理标准是使用IX因子（FIX）产品进行预防。该分析评估了德国和意大利接受rIX-FP或rFIXFc预防的B型血友病（PwHB）患者的预防效果。方法：回顾性、去识别的图表回顾包括来自德国或意大利的PwHB≥12年的重度/中度血友病B，接受rIX-FP或rFIXFc预防治疗≥12个月。主要终点是FIX的消耗；次要结果为给药间隔、年化出血率（ABR）、年化自发出血率（AsBR）和年化关节出血率（AjBR）。这些结果也通过rix - fp前后开关数据在PwHB中进行了探讨。结果：194例PwHB患者中，分别有107例和87例接受了rIX-FP和rFIXFc预防。rIX-FP的平均FIX消耗量显著低于rFIXFc (42.4 vs. 65.2 IU/kg/week, p = 0.0001)，平均给药间隔为9.5天（rIX-FP）和7.9天（rFIXFc）。rIX-FP与rFIXFc的平均出血率分别为：ABR 0.7 vs 1.1 (p = 0.6704), AsBR 0.1 vs 0.3 (p = 0.3427), AjBR 0.3 vs 0.4 （p = 0.5296）。在比较这些结果时，PwHB合并重度和中度血友病B的亚组分析分别显示出相似的数值模式。在18例具有切换数据的患者中，观察到FIX消耗显著减少（中位51.7至33.3 IU/kg/周，p = 0.0069），平均给药间隔延长（7.2-9.5天）。ABR(中位数1.6-0.0,p = 0.0172；n = 18)和AjBR(中位数0.6-0.0,p = 0.0200；n = 14)显著降低，而AsBR降低但不显著(中位数0.2-0.0,p = 0.1460；n = 14)。结论：与rFIXFc相比，rIX-FP预防与FIX消耗减少相关，并提供同样有效或潜在改善的出血保护。此外，与之前的FIX产品相比，改用rIX-FP的PwHB在FIX消耗、ABR和AjBR方面都有显著降低。
Real-World Prophylaxis Outcomes with rIX-FP and rFIXFc for Males with Hemophilia B: Pooled Analysis of Medical Chart Data from Germany and Italy
Introduction
The current standard of care for people with severe hemophilia B is prophylaxis with factor IX (FIX) products. This analysis assessed the effectiveness of prophylaxis for people with hemophilia B (PwHB) receiving rIX-FP or rFIXFc prophylaxis in Germany and Italy.
Methods
A retrospective, de-identified chart review included PwHB ≥ 12 years with severe/moderate hemophilia B from Germany or Italy, receiving prophylaxis with rIX-FP or rFIXFc for ≥ 12 months. The primary outcome was FIX consumption; the secondary outcomes were dosing interval, annualized bleeding rate (ABR), annualized spontaneous bleeding rate (AsBR), and annualized joint bleeding rate (AjBR). These outcomes were also explored in PwHB with pre- and post-rIX-FP switch data.
Results
Of 194 PwHB, 107 and 87 received rIX-FP and rFIXFc prophylaxis, respectively. The mean FIX consumption of rIX-FP was significantly lower compared to rFIXFc (42.4 vs. 65.2 IU/kg/week, p = 0.0001), with mean dosing intervals of 9.5 days (rIX-FP) and 7.9 days (rFIXFc). The mean bleeding rates for rIX-FP versus rFIXFc, respectively, were: ABR 0.7 versus 1.1 (p = 0.6704), AsBR 0.1 versus 0.3 (p = 0.3427), and AjBR 0.3 versus 0.4 (p = 0.5296). Subgroup analyses for PwHB with severe and moderate hemophilia B separately showed similar numerical patterns when comparing these outcomes. In the 18 patients with switch data, a significant reduction in FIX consumption was observed (median 51.7 to 33.3 IU/kg/week, p = 0.0069), and the mean dosing interval was extended (7.2–9.5 days). The ABR (median 1.6–0.0, p = 0.0172; n = 18) and AjBR (median 0.6–0.0, p = 0.0200; n = 14) decreased significantly, while the AsBR decreased but not significantly (median 0.2–0.0, p = 0.1460; n = 14).
Conclusion
rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product.
期刊介绍：
Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged.
The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.
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